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BT910

Development stage: Preclinical
Lead developer: Base Therapeutics
Modality: CRISPR-Cas9 → CRISPR Systems → Programmable Nucleases → Gene Editing → Gene Therapies
Administration: Ophthalmic

Overview

BT910 is an investigational gene therapy being developed by **Base Therapeutics** for the treatment of **Age-related Macular Degeneration (AMD)**. It utilizes the company's proprietary **AccuBase** base editing technology platform to perform precise genetic modifications. Unlike traditional CRISPR-Cas9 systems that create double-strand breaks, base editing allows for single-nucleotide changes without breaking the DNA backbone, which may offer a improved safety profile for ocular applications. While the specific molecular target for BT910 has not been publicly disclosed, the program is currently in the IND-enabling stage of development.

Targets

gDNA (Genomic DNA at disease-specific locus)

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